An engineered truncated gene derived from the dystrophin-related protein (utrophin), prevents pathology without an immune response in an animal model of Duchenne muscular dystrophy (DMD) gene therapy.
This is a preview of subscription content, access via your institution
Relevant articles
Open Access articles citing this article.
-
Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders
Neurological Research and Practice Open Access 04 January 2022
-
Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy
European Journal of Human Genetics Open Access 09 February 2021
-
Muscle spindle function in healthy and diseased muscle
Skeletal Muscle Open Access 07 January 2021
Access options
Access Nature and 54 other Nature Portfolio journals
Get Nature+, our best-value online-access subscription
$29.99 / 30 days
cancel any time
Subscribe to this journal
Receive 12 print issues and online access
$209.00 per year
only $17.42 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
References
Guiraud, S. et al. The pathogenesis and therapy of muscular dystrophies. Annu. Rev. Genomics Hum. Genet. 16, 281–308 (2015).
Song, Y. et al. Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models. Nat. Med. https://doi.org/10.1038/s41591-019-0594-0 (2019).
Duan, D. Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy. Mol. Ther. 26, 2337–2356 (2018).
Chamberlain, J. R. & Chamberlain, J. S. Progress toward gene therapy for Duchenne muscular dystrophy. Mol. Ther. 25, 1125–1131 (2017).
Mendell, J. R. et al. Brief report: dystrophin immunity in Duchenne’s muscular dystrophy. N. Engl. J. Med. 363, 1429–1437 (2010).
Guiraud, S. & Davies, K. E. Pharmacological advances for treatment in Duchenne muscular dystrophy. Curr. Opin. Pharmacol. 34, 36–48 (2017).
Harper, S. Q. et al. Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. Nat. Med. 8, 253–261 (2002).
Odom, G. L. et al. Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice. Mol. Ther. 16, 1539–1545 (2008).
Kennedy, T. L. et al. Micro-utrophin improves cardiac and skeletal muscle function of severely affected D2/mdx mice. Mol. Ther. Methods Clin. Dev. 11, 92–105 (2018).
Ramos, J. N. et al. Development of novel micro-dystrophins with enhanced functionality. Mol. Ther. 27, 623–635 (2019).
Crudele, J. M. & Chamberlain, J. S. AAV-based gene therapies for the muscular dystrophies. Hum. Mol. Genet. https://doi.org/10.1093/hmg/ddz128 (2019).
Guiraud, S. et al. The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy. Hum. Mol. Genet. 28, 2189–2200 (2019).
Author information
Authors and Affiliations
Corresponding author
Ethics declarations
Competing interests
J.S.C. is a member of the Scientific Advisory Board for Solid Biosciences. K.E.D. has no declared conflicts.
Rights and permissions
About this article
Cite this article
Davies, K.E., Chamberlain, J.S. Surrogate gene therapy for muscular dystrophy. Nat Med 25, 1473–1474 (2019). https://doi.org/10.1038/s41591-019-0604-2
Published:
Issue Date:
DOI: https://doi.org/10.1038/s41591-019-0604-2
This article is cited by
-
Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders
Neurological Research and Practice (2022)
-
Muscle spindle function in healthy and diseased muscle
Skeletal Muscle (2021)
-
Evaluating the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy
European Journal of Human Genetics (2021)